The Bytes of Cancer

ISSN 2364-2351 | A 6 0 711 | Interview José Pfizer, IP Head of 4SC AG, explains Life Sciences and how innovative Industry Magazine SMEs perceive the EU’s unitary Autumn Edition 2017 | Volume 16 | 20 € patent

FREE EXCERPT Big Data War The Bytes of Cancer

Neurodegeneration Archaea Bioeconomy Biomanufacturing Drug developers are hunting for Are these extremophiles the Insect protein – the Next Latest R&D and packaging a cure to Huntington’s disease solution to the energy crisis? Big Thing in food and feed? trends for CROs, CMOs & CPOs YOUR MODULAR

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www.polpharmabiologics.com COMMERCIAL GMP MANUFACTURING [email protected] 4 Contents European Biotechnology | Autumn Edition | Vol. 16 | 2017

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Cover Story Insight Europe regional news

6 Germany postpones ratification 46 northern Europe: process for European Unitary Sweden, Denmark, Finland Patent and Unitary Patent Court until after Brexit 48 Western Europe: France, Belgium, The Netherlands 10 Interview: “SME Perspective on and the UK the Unitary Patent” Dr. Jose Pfizer, Head of Intellectual Property, 50 Central Europe: 4SC AG, Martinsried Germany, Switzerland and Austria 12 Biofuels: New study shows EU policymaker’s “tank or table” 54 Southern Europe: paradigm isn’t based on facts Italy, Spain and Portugal

14 eU Inmare project/Bioeconomy: 56 eastern Europe: Diving for industrial enzymes Poland, Czech Republic, Slovak Republic and Estonia 15 e uropean Biotech Week showcases biotech and bio-based Science & Technology solutions to MEPs 90 Bioeconomy: Insect protein 16 e xpert comment: producers target food and feed 18 Germany’s freeze of the markets in Europe Unified Patent Court, Dr. Ute Kilger, Boehmert & Boehmert 92 Untangling neurodegeneration; AI and cancer – the Cancer drug from mahagony tree; Economy Refurbished Parkinson’s drug; modern data wars Halting tumour escape 28 novartis AG’s CAR-T success to Big Data is changing the way doctors be discussed at BIO-Europe 92 Swiss researchers find long-sought make decisions, and redefining priva- source of neurotoxicity in cy. Should companies be allowed to 29 Update on clinical trials Alzheimer’s disease lock up patient information in proprietary databases? Or should it be open ac- 32 What’s on at BIO-Europe in Berlin Service cess? Does medical data like a tumour’s DNA sequence belong to the patient, or 35 Analyst commentary: 81 news from partner associations: should it be pseudonymised for use in Samir Devani, Managing Director, SBA, BIO Deutschland, DIA, studies aimed at curing the disease? And Rx Securities EuropaBio, EBN not least – how quickly will cognitive systems really make a difference in out- 36 Euro Biotech Stocks 105 Company index comes? A lot of money is at stake in the 104 Events field, as well as people’s lives. 89 EMA News 106 Encore

IMPRINT European Biotechnology (ISSN 2364-2351) is published quarterly by: BIOCOM AG, Lützowstr. 33–36, D-10785 Berlin, Germany, Tel.: +49-30-264921-0, Fax: +49-30-264921-11, Email: [email protected], Internet: www.european-biotechnology.com; Publisher: Andreas Mietzsch; Editorial Team: Thomas Gabrielczyk (Editor in Chief), Derrick Williams (Co-editor), Uta Mommert, Dr. Martin Laqua, Sascha Karberg, Helene Märzhäuser; Advertising: Oliver Schnell, +49-30-2649-2145, Christian Böhm, +49-30-2649-2149, Andreas Macht, +49-30-2649-2154; Distribution: Marcus Laschke, +49-30-2649-2148; Graphic Design: Oliver-Sven Reblin; Production editor: Benjamin Röbig; Printed at: Königsdruck, Berlin; European Biotechnology Life Sciences & Industry Magazine is only regularly available through subscription at BIOCOM AG. Annual subscription fees: € 80.00, Students € 40.00 (subject to proof of enrolment). Prices include VAT, postage & packaging. Ordered subscriptions can be cancelled within two weeks directly at BIOCOM AG. The subscription is initially valid for one year. Subscriptions will be renewed automatically for one more year, respectively, unless they are cancelled at least six weeks before the date of expiry. Failures of delivery, which BIOCOM AG is not responsible for, do not entitle the subscriber to delivery or reimbursement of pre-paid fees. Seat of court is Berlin, Germany. As regards contents: individually named articles are published within the sole responsibility of their respective authors. All material published is protected by copyright. No article or part thereof may be reproduced in any way or processed, copied and proliferated by electronic means without the prior written consent of the

publisher. Cover Photo: © Kotkoa/fotolia.com; Supplement: Bähren Druck, IZB, ® BIOCOM is a registered trademark of BIOCOM AG, Berlin, Germany. Picture: publicdomainpictures.net CC1.0 European Biotechnology | Autumn Edition | Vol. 16 | 2017 Contents 5

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Neurodegeneration Bioeconomy Editorial

Curing Huntington’s Digital reshape Digitalisation is set to become the It slowly but relentlessly destroys your buzzword of 2017. Whether you’re body and your mind, and there’s nothing talking about patient selection for clin- doctors can do to stop it. At least, until ical trials, cancer therapy decision sup- now. 25 years after an American scien- port, customer relations, direct-to-con- tist discovered the genetic mutation that sumer advertising or even supply causes Huntington’s disease, medical re- chain management – everyone is con- search is finally beginning to catch up. vinced digitalisation is the key to effi- An ongoing trial is attempting to stop the ciency. For some, however, the word disease in its tracks. And one day a gene is an empty one. Recently one Ger- therapy solution might effectively cure it. man biotech CEO stressed that “it’s just a tool, not the solution”. In other words, don’t expect it to cure cancer. On p.18, our cover story shows what 94 kind of value creation digital data from cancer mutations and treatment out- comes could contribute in the long Green methane run to more personalised therapies. Although there’s hype and PR in over- They live in some of the most extreme en- selling computational correlation vironments on Earth, including hydrother- analyses, researchers from companies mal vents and geysers. Archaea are fasci- and hospitals agree that it will one day nating microorganisms that seem to have improve prognosis and decision sup- changed little since the dawn of life. Now port. A big issue, though, is access to they could help us solve one of our most results. 40 pressing modern problems – how to store Looking forward to the CPHI and excess power from renewable sources. BIO-Europe, we’ve addressed the top- ics that are most relevant to the sector, among them the EU unitary patent (see p. 6–8), the latest outsourcing trends in biomanufacturing, pharma packaging, SPECIAL and contract research, along with the latest news from the growing bioeconomy sector. A new EU strategy is being CROs, CPOs & CMOs drafted (p.15) while EU companies are working to replace GM soy protein 59 Prepared for next-gen biologics with insect-based feed (p. 90). Enjoy! 64 Polpharma Biologics: expanding scale and reach 66 Insight into Biomarkers 68 Advanced formulations for biologics 74 Interview: Aldo Poli, CEO, Opis 76 Need a contemporary manu facturing strategy? 79 Rapid implementation of off-line serialisation 83 Serialisation using labels Thomas 84 Injection pens in clinical trials Gabrielczyk 86 New products Editor-in-Chief Pictures: Wikimedia Commons/James St. John (top), visualspace /istockphoto.com (middle), Adents (bottom) 18 Rubrik European Biotechnology | Spring Edition | Vol. 16 | 2017

Finding driver mutation patterns in tumour genomes could turn the fields of AI/bioinformatics into a stethoscope for st the 21 Century Pictures: xxx European Biotechnology | Autumn Edition | Vol. 16 | 2017 Cover Story 19

FREE EXCERPT Cancer data: A dive into the depths

Correlation Sciences As Big Data analytics begin to inspire medical decisionmaking, companies and scientists have gone to war over access to data derived from patient biopsies. Cancer profiling is a seminal situation. While some companies are seeking to profit from approaches to advanced mutation analysis, researchers are vociferous in demanding open access to proprietary databases behind company firewalls.

ccording to William – “call based pricing by limiting reimbursement vogue in the sector. In 2013, IBM began me Bill” – Kassler, human de- of costly targeted cancer therapies or promoting Watson as a ‘best treatment’ cisions are anything but per- immuno-oncological treatments to pa- detector. The claim was based on min- fect. “But machines can de- tient groups that have a proven bene- ing cancer genomes and corresponding bugA that,” stresses the Deputy Chief fit. It’s no secret that current Compan- treatment data stored in Electronic Medi- Health Officer & Lead Population Health ion Diagnostics (CDx) are not designed cal Records (EMRs). According to Kassler, Officer at IBM Watson Health in Boston. for cancer mutation pattern detection. Watson’s learning algorithms – which he Speaking last summer at the Forum Sci- But because cancer-promoting effects brands “artificial intelligence” – could as- ence & Health conference in Munich, the are dependent on surrounding genes sist physicians on two fronts: ex-CDC policy advisor and former Medi- and an individual genomic context, AI- care expert for value-based drug pric- directed genome mining is currently en ›› collecting and interpreting the flood ing predicted that cognitive computers of medical discoveries from papers, would be the Next Big Thing in personal- slides, clinical trials, and other data ised medicine. Intelligent machines such ›› finding unrecognised patterns in real as IBM Watson, he insisted, could learn world data, including relevant muta- rapidly. “Within just four years, Watson tions in cancer genomes, prognos- scaled down its error rate from over 40% tic patterns in EMRs, images, lifestyle to 5% in recognising language. And that’s data from fitbits, etc. just the beginning,” says Kassler. What if that power was turned to unravelling hid- Experts like Roger Schank say that den patterns in tumour genomes? Watson simply “correlates data”, and that this ability is subsequently sold as ‘AI’ for Why data analytics could Josh Lauer Lifecycle Leader, marketing purposes. He stresses that an transform biopharma Foundation Medicine (FMI) at Roche ability to sift through a large amount of data doesn’t define intelligence. The da- The first applications for these kinds of How can Roche help to expand ta-mining approach, however, has at- learning machines and algorithms are ? FMI’s capabilities? tracted Big Data analytics majors like currently surfacing in personalised med- IBM, Google, its spin-out Verily, Micro- icine, particularly in the highly lucra- We have to build on FMI interna- soft and a growing number of high-tech tive field of cancer. Kassler knows that ! tional traction by leveraging on SMEs. That’s because it can generate ex- identifying drug responders to a growing presence in many countries. We actly the data needed by payors to […] number of cancer combination therapies have already launched the service and immunotherapies is still quite lim- in 12 countries. We look forward to ›› Read the full story in the printed issue. ited. He also knows that cash-strapped bringing it to every market.

Pictures: Roche (right hand side), publicdomainpictures.net CC1.0health (left) systems want to switch to value 40 Neurodegeneration European Biotechnology | Autumn Edition | Vol. 16 | 2017 FREE EXCERPT

The hunt for a cure to Huntington’s

Genetic Diseases It’s a disease that often strikes people in the prime of life, slowly destroying control over body, mind and personality. It inevitably kills the patient, but usually only after at least a decade of suffering. The underlying cause of Huntington’s disease – a dominant mutation on Chromosome IV – was discovered almost 25 years ago. Now the first trials to target the condition at its source have begun. Chances of finding a cure have never been more realistic.

›› Read the full story in the printed issue. Picture: visualspace /istockphoto.com 94 BioEconomy European Biotechnology | Autumn Edition | Vol. 16 | 2017

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Archaea were first discovered in extreme environments like hot springs. Depicted is the Grand Prismatic Spring in Yellowstone National Park in the US.

Hot guys in the pool rescue sustainability

Biological Methanation The renewable energy sector is ramping up in a major way, but a host of infrastructure issues still need to be solved. One is that supply at times now outstrips demand, but there’s no way to store excess power. Turning it into an easy-to-store, energy-rich source like methane could solve the problem. A pivotal aspect of ‘power-to-gas’ technology harnesses some tiny helpers usually found in some of the most extreme regions on the planet.

›› Read the full story in the printed issue. Picture: Wikimedia Commons/James St. John 104 company index European Biotechnology | Autumn Edition | Vol. 16 | 2017

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23&me (USA)...... 20 Galapagos NV (B)...... 29 Oxford BioMedica plc (UK)...... 35, 50 3P Biopharmaceuticals S.L. (ES)...... 54, 72 Genentech Inc. (USA)...... 30 Palantir Technologies Inc. (USA)...... 20 4SC AG (GER)...... 10 GeNeuro S.A. (CH)...... 35 PCI Biotech AS (N)...... 47 Genmab A/S (DK)...... 30, 46 Peppermint Financial Partners (GER)...... 51 Ablynx BV (B)...... 35, 48 GenPat 77 Pharmacogenetics AG (GER)...... 51 Pfizer (USA)...... 35, 47 Actelion AG (CH)...... 35 GenSearch Consulting (GER) ...... 7 Pfizer CentreOne (USA)...... CP4 AdrenoMed AG (GER) ...... 29, 30 Gensoric (GER)...... 97 PharmaMar S.A. (ES)...... 14, 30 Aglaia BioMedical Ventures B.V. (NL). . . . . 49 Gilead Sciences Internation Ltd. (USA). . . 35, 89 Pieris Pharmaceuticals Inc. (USA)...... 28 Amsterdam Molecular Therapeutics (NL) ...... 48 GlaxoSmithKline (UK) ...... 48 polpharma biologics (PL)...... CP2, 64 Aptuit LLC (USA) ...... 35 Google Inc. (USA) ...... 19, 24 Promocell GmbH (GER)...... 68 Ascletis Bioscience Co. Ltd (CN)...... 47 ProScience Polska/BioInnovation Int. Summit. . 55 ASIT biotech (B)...... 31 Hansa Medical AB (SE)...... 35, 46 Proti-Farm (NL)...... 91 AstraZeneca AB (SE/UK)...... 20, 48 Hermetia (GER) ...... 91 Protix BV (NL) ...... 91 Axxam SpA (IT)...... 54 High Tech Gr√ºnderfonds GmbH (GER) ...... 51 AyoxxA Biosystems GmbH (GER). . . . . 66, 71 HiProMine SA (PL)...... 91 Rentschler Biotechnologie GmbH (GER). . .65, 68 Hitgen Ltd. (CN)...... 47 Richter-Helm BioLogics GmbH & Co. KG (GER). 87 Bardehle Pagenberg (GER) ...... 8 Rigontec GmbH (GER)...... 51 Basilea Pharmaceutica (CH)...... 35 I&L Biosystems GmbH (GER)...... 21 Roche AG (CH)...... 19, 20, 29 Bavarian Nordic A/S (DK)...... 35, 46 IBM Corp. (USA)...... 19, 24 Roche Diagnostics Deutschland GmbH...... 58 Bayer AG (GER) ...... 14, 29, 35 Illumina Inc. (USA)...... 20 Roivant Sciences GmbH (CH)...... 51 Berlin Partner GmbH (GER) ...... 38, 39 Incyte Corporation (USA)...... 28 Rondo AG (CH) ...... 79, 80, 81, 85 Berlin-Chemie Menarini (GER)...... 54 INOFEA Ltd. (CH)...... 14 Rx Securities (UK)...... 35 Beta Biotech (SK) ...... 54 Ionis Pharmaceuticals (USA)...... 42, 45 BIOCOM AG (GER) ...... 15, 61, 103, CP3 SalvaRx Group (UK)...... 51 Biontech AG (GER)...... 30 Janssen Biotech Inc. (USA)...... 20, 46 Sanofi SA (F)...... 20, 35, 48, 54 Boehmert & Boehmert (GER) ...... 16 Janssen Pharmaceutica N.V. (NL)...... 31, 35, 47 Sanofi-Genzyme (USA)...... 44, 45 Boehringer Ingelheim (GER)...... 29 Johnson & Johnson (USA)...... 35, 46 Santhera Pharmaceuticals (CH)...... 50 Boehringer Ingelheim Venture Fund (GER). . . . . 51 Selvita sp. z o.o. (PL) ...... 54 Bravo Pharmaceuticals (IN)...... 54 Kiadis Pharma BV (NL)...... 36 Sobi AB (SE)...... 35 Bristol Laboratories Ltd. (UK)...... 46 Kite Pharma (USA)...... 35 Softbank Vision Fund (JP) ...... 51 Bristol Myers Squibb (USA)...... 29, 31 Sophia Genetics S.A. (CH)...... 20, 51 Bryan, Garnier & Co. (F)...... 35 LEO Pharma A/S (DK)...... 47 Spark Therapeutics (USA)...... 48 Bühler Insect Technology Solutions (CH) ...... 91 Les Laboratoires Servier S.A. (F) ...... 28 Summit Therapeutics plc (IRL)...... 35 Leukocare AG (GER)...... 68 Sunstone Capital (DK)...... 51 CANDOR Bioscience GmbH (GER)...... 86 Life Science Austria LISA ...... 25 Swiss Biotech Association (CH)...... 88 Cellact Pharma GmbH (GER)...... 51 Lipotype GmbH (GER)...... 38, 39 Cellectis SA (F)...... 28 Thermo Fisher Scientific (UK)...... 22, 24 Celonic AG (CH) ...... 62, 63 M+W Central Europe GmbH (GER) ...... 73 ThromboGenics NV (B) ...... 49 Celyad SA (F)...... 28 Macrophage Pharma Ltd (UK)...... 49 Tiziana Life Sciences plc (UK)...... 35 Charles River (USA) ...... 77 MediGene (GER) ...... 28 Topas Therapeutics GmbH...... 51 Chiesi Farmaceutici S.p.A. (IT)...... 48 Medivir AB (SE)...... 30, 47 Transgene SA (F)...... 31 Chroma Therapeutics Ltd. (UK)...... 49 Merck & Co. (USA)...... 49, 51 Trianni, Inc. (USA) ...... 23 CureVac AG – mRNA Conference 2017 (GER). . 53 Merck KGaA (GER)...... 20, 22, 24, 47 Merus B.V. (NL) ...... 28 UCB S.A. (B)...... 29 DECHEMA Ausstellungs-GmbH (GER)...... 25 Metsäs Group Coop. (FI)...... 47 UGA Biopharma GmbH (GER)...... 86 Deutsche Bank AG (GER)...... 35 MicrobEnergy GmbH (GER)...... 96 uniQure BV (NL) ...... 48 Dexxon Investor Group (IL) ...... 51 Micronutics (F)...... 91 MicroPyros GmbH (GER)...... 96 Valneva (F/A) ...... 51 EBD Group (GER)...... 28, 32, 33 Microsoft Corp. (USA) ...... 19, 24 Vectron Biosolutions AS (N)...... 69 Elektrochaea GmbH (GER)...... 95, 96 MLM Medical Labs GmbH (GER)...... 11 Veeva Systems Inc (USA) ...... 3 Eli Lilly & Co Ltd (USA) ...... 51 Mologen AG (GER)...... 31 Verily Life Scienes (USA) ...... 19, 20 Emergent Biosolutions (USA) ...... 51 MorphoSys AG (GER)...... 29 Verona Pharma plc (UK)...... 106 Entomo Farm (F)...... 91 MP Healthcare Venture Management (USA). . . . 51 Vesalius Biocapital Partners S.a.r.L. (LU). . . . 54 Eppendorf AG/Bioprocess Center Europe (GER) . 43 Mundipharma EDO GmbH (CH) ...... 51 Vetter Pharma-Fertigung GmbH (GER). . 31, 76, 78 EuropaBio (B)...... 9 Myriad Genetics (USA)...... 22 VIB Discovery Sciences (B)...... 49 evocatal GmbH (GER) ...... 14 Viessmann GmbH (GER)...... 96 Evotec AG (GER)...... 35, 51 Nekonal Oncology (LU)...... 51 Viking Global Investors (USA)...... 51 NextAlim (F)...... 91 Voyager Therapeutics (USA)...... 45 Fair Insects BV (NL) ...... 91 Nordea Bank (DK) ...... 35 VTU Technology GmbH (AT) ...... 81 Faron Pharmaceuticals Oy (FI)...... 35 nova-Institut GmbH (GER) ...... 15 FGK Clinical Research GmbH (GER) ...... 78 Novartis AG (CH)...... 8, 20, 28, 35, 50 Wellington Partners (GER)...... 51 FibroGen Inc. (USA)...... 29 Novo Holdings A/S (DK)...... 49 Wilhelm Bähren GmbH (GER). . . . 83, Supplement Flatiron Health (USA)...... 20 Novozymes A/S (DK) ...... 14 Wilhelm Haselmeier GmbH (GER). . . . .83, 84 Focus First (USA) ...... 95 NRW Bank (GER)...... 51 Fördergesellschaft IZB (GER). . . . . Supplement Xell AG (GER)...... 75 Forbion Capital Partners (NL) ...... 51 Oncurious NV (B)...... 49 Foundation Medicine Inc. (USA)...... 20, 24 OPIS s.r.l. (IT)...... 29, 74 Ynsect (F)...... 90 Freenome (USA)...... 20 Orion Corporation (FI)...... 61 FUJIFILM Diosynth Biotechnologies (USA). . . 67 ORPEGEN Peptide Chemicals GmbH (GER). . .34 Zealand Pharmaceuticals A/S (DK)...... 31, 47 UI#FSMJO$POGFSFODFPO-JGF4DJFODFT /PWFM"OUJNJDSPCJBMT BOE".3%JBHOPTUJDT

Picture: Dimitar Marinov / fotolia.com / Dimitar Marinov Picture: 2 March 2018, Berlin, Germany

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